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| Sponsor: | Columbia University |
|---|---|
| Information provided by: | Columbia University |
| ClinicalTrials.gov Identifier: | NCT00881556 |
Purpose
Reduced Intensity Conditioning (RIC) and Allogeneic Stem Cell Transplantation (AlloSCT) from family-related donors and unrelated cord blood (UCB) donors will be safe and well tolerated in selected patients with RDEB.
To determine the event-free survival (EFS) and overall survival (OS) following RIC consisting of busulfan/fludarabine/alemtuzumab (BFA) and AlloSCT in selected patients with RDEB.
| Condition | Intervention | Phase |
|---|---|---|
|
Epidermolysis Bullosa |
Drug: Reduced Intensity Transplant conditioning |
Phase 0 |
| Study Type: | Interventional |
| Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
| Official Title: | A Pilot Study of Reduced Intensity Conditioning (RIC) and Allogeneic Stem Cell Transplantation (ALLOSCT) In Children With Recessive Dystrophic Epidermolysis Bullosa (RDEB) |
| Estimated Enrollment: | 20 |
| Study Start Date: | March 2009 |
| Estimated Study Completion Date: | July 2014 |
| Estimated Primary Completion Date: | June 2014 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
1: Experimental
Reduced Intensity
|
Drug: Reduced Intensity Transplant conditioning
Palifermin (Kepivance®) 60 mcg/kg/day for 6 days Fludarabine 30 mg/m2 IV x 1 for 6 days Busulfan 4 mg/kg/day IV divided BID for 4 days Lorazepam 0.02-0.05 mg/kg for 5 days Alemtuzumab 20 mg/m2 IV for 5 days Tacrolimus 0.03mg/kg/24 hours as continuous infusion for 4 days |
Epidermolysis bullosa (EB), is a diverse group of genodermatoses, which is considered a rare and orphan disease and affects approximately 1 in 20,000 people in the United States for a cumulative total of close to 20,000[1-4]. There are three major subtypes of inherited EB, including EB simplex (EBS), junctional EB (JEB), and dystrophic EB[1-4]. RDEB is among the most severe and represents approximately 10% of all forms of EB[1-4]. A rough estimate would then project that there are several thousand patients with RDEB in the U.S. at the current time. Up to 30 different clinical phenotypes and mutations in at least 10 structural genes in different sub-types of EB have been reported[4-8]. In addition to heritable subtypes of EB, there is an acquired autoimmune form in which the patients develop auto-antibodies directed against similar proteins of the inherited dystrophic forms of EB, including EB acquisita (EBA).
We have previously reported our experience with RIC with BFA [48] in pediatric AlloSCT recipients (mean age 9.5 yrs [1.4-21], 11/4 M/F, 10 non-malignant, 5 malignant disease, [6 sibling, 5 UCB, 5 matched unrelated donor]); median time to ANC ≥ 500/mm3 and platelet count ≥20K/mm3 was 22 and 30 days, respectively. Probability of day +180 and 365 donor chimerism was 90% (Figure 7), and OS was 95% (Figure 8). This conditioning regimen therefore results in a high degree of donor chimerism and survival with minimal regimen related mortality.
Eligibility| Ages Eligible for Study: | up to 21 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Patient must have adequate organ function as below:
Adequate renal function defined as:
Adequate liver function defined as:
Adequate cardiac function defined as:
Adequate pulmonary function defined as:
Exclusion Criteria:
Contacts and Locations| Contact: Mitchell S Cairo, MD | 212-305-8316 | mc1310@columbia.edu |
| Contact: Claire Fanelli, RN | 212-305-2050 | cf2370@columbia.edu |
| United States, New York | |
| Morgan Stanley Children's Hospital of NYP | Recruiting |
| New York, New York, United States, 10032 | |
| Contact: Mitchell S Cairo, MD 212-305-8316 mc1310@columbia.edu | |
| Contact: Claire Fanelli, RN 212-305-2050 cf2370@columbia.edu | |
| Principal Investigator: Mitchell S Cairo, MD | |
| Principal Investigator: | Mitchell S Cairo, MD | Columbia University |
More Information
| Responsible Party: | Columbia University Medical Center ( Mitchell S. Cairo MD ) |
| Study ID Numbers: | AAAD5420, CHNY-08-536 |
| Study First Received: | April 14, 2009 |
| Last Updated: | April 23, 2009 |
| ClinicalTrials.gov Identifier: | NCT00881556 History of Changes |
| Health Authority: | United States: Institutional Review Board |
|
Allogeneic Stem Cell Transplant AlloSCT RDEB recessive dystrophic epidermolysis bullosa |
|
Collagen Diseases Skin Diseases, Vesiculobullous Skin Diseases Genetic Diseases, Inborn Connective Tissue Diseases |
Skin Abnormalities Epidermolysis Bullosa Dystrophica Epidermolysis Bullosa Congenital Abnormalities Skin Diseases, Genetic |
